Summary

Eligibility
for people ages 2-53 (full criteria)
Location
at Oakland, California and other locations
Dates
study started
estimated completion

Description

Summary

This is a multi-center, long-term safety and efficacy follow-up study for subjects with sickle cell disease who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies. After completing the parent clinical study (approximately 2 years), eligible subjects will be followed for an additional 13 years for a total of 15 years post-drug product infusion. No investigational drug product will be administered in the study.

Official Title

Long-term Follow-up of Subjects With Sickle Cell Disease Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector

Keywords

Sickle Cell Disease Hematopoietic Stem Cells Anemia, Sickle Cell Safety and efficacy assessments

Eligibility

You can join if…

Open to people ages 2-53

  • Provision of written informed consent for this study by subject, or as applicable, subject's parent(s)/legal guardian(s)
  • Treated with drug product for therapy of sickle cell disease in a bluebird bio-sponsored clinical study

You CAN'T join if...

  • There are no exclusion criteria for this study

Locations

  • UCSF Benioff Children's Hospital Oakland
    Oakland California 94609 United States
  • Ann & Robert H. Lurie Children's Hospital of Chicago
    Chicago Illinois 60611-2991 United States

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
bluebird bio
ID
NCT04628585
Study Type
Observational
Participants
Expecting 85 study participants
Last Updated